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Yale Physician-Scientists Pioneer Fetal Gene Therapy for Cystic Fibrosis

David Stitelman, MD, has received funding from the Cystic Fibrosis Research Institute to advance his collaborative research on a novel fetal therapy approach for cystic fibrosis. Working with colleagues across pediatric pulmonology, biomedical engineering, and gene editing, his team is developing nanoparticle-based strategies to deliver gene therapies before birth—aimed at preventing organ damage that begins in utero. By combining mRNA delivery and high-fidelity gene editing, this work has the potential to move toward a one-time treatment for cystic fibrosis and establish a new paradigm for treating genetic diseases before birth.

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